OBJECTIVES: PNH is a rare, acquired, life-threatening, chronic hematologic disease caused by terminal complement activation on the surface of all blood cells. The introduction of the first C5-inhibitor, eculizumab, has substantially improved the survival and quality of life of PNH patients. In 2019, C5-inhibitor ravulizumab was approved by the European Medicine Agency. Due to its engineering, C5-inhibition ravulizumab requires an 8-weekly infusion instead of biweekly for eculizumab. This study aims to evaluate the cost-effectiveness of ravulizumab compared with eculizumab in adult patients in the Netherlands with PNH and clinical symptom(s) indicative of high disease activity or who were treated with eculizumab for at least six months and are clinically stable.

METHODS: A Markov cohort model was developed to describe the course of PNH and subsequently model the costs and quality-adjusted life-years (QALYs) associated with both C5-inhibitors from a Dutch societal perspective. Health states included no breakthrough hemolysis (BTH), BTH, spontaneous remission, and death. Medical (drug, administration, transfusion, BTH related) and societal (travel and productivity) costs, QALYs, and incremental cost-effectiveness ratio (ICER) were estimated over a lifetime, using a discount rate of 1.5% in line with the Dutch health economics guidance.

RESULTS: Due to its reduced administration frequency and fewer BTH events, ravulizumab was associated with lower costs (-€266,833) and a gain of 1.57 QALYs compared to eculizumab, resulting in a dominant ICER. Drug costs were the main cost drivers in both treatment, with the difference leading to a reduction of €260,587 (-4.5%) in favor of ravulizumab. Additionally, compared to eculizumab, ravulizumab reduced medical and societal costs by €1,578 and €4,699 respectively. The probability of ravulizumab being cost-effective at a willingness-to-pay of €20,000/QALY was 76.6%.

CONCLUSIONS: Compared to eculizumab, ravulizumab is a dominant treatment strategy and is associated with an improved quality of life for adults with PNH in the Netherlands.